A New Intergenic α -Globin Deletion ( α – α Δ125 ) Found in a Kabyle Population

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Two Tumors in 1: What Should be the Therapeutic Target? Pediatric Germ Cell Tumor With Somatic Malignant Transformation

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Extracranial germ cell tumours in children and adolescents: Results from the French TGM13 protocol

International audienceBackground: Chemotherapy for non-seminomatous germ cell tumours (NSGCT) exposes to dose-dependent toxicities. The TGM13-NS protocol (EudraCT 2013-004039-60) aimed to decrease the chemotherapy burden compared to the previous TGM95 protocol while maintaining the 5-year event-free survival (EFS) at 80% or more. Procedure: Patients less than 19 years of age with disseminated NSGCT were enrolled (May 2014 to May 2019) and stratified into four groups: two intermediate-risk (IR: localised tumour with low tumour markers [TM]) groups treated with VBP (vinblastine–bleomycin–cisplatin): three courses for IR1 (ovarian tumour any age/testis tumour less than or equal to 10 years) and four courses for IR2 (extragonadal tumour 10 years or less) groups, and two high-risk (HR: metastatic and/or high TM) groups treated with etoposide–cisplatin and either ifosfamide (VIP) or bleomycin (BEP): three courses for HR1 (ovarian tumour any age/testis tumour less than or equal to 10 years and low TM/testis tumour more than 10 years and very low TM) groups and four courses for HR2 (remainder) groups. Results: One hundred fifteen patients were included: median age of 12.8 years (0.4–18.9); tumour sites: 44 ovaries, 37 testes and 34 extragonadal. The 5-year EFS and overall survival (OS) were 87% (95% CI: 80–92) and 95% (89–98), respectively (median follow-up: 3.5 years, range: 0.2–5.9), similar to those of the TGM95 protocol (5-year EFS 89% (84–93), 5-year OS 93% (89–95), p =.561). The 5-year EFS were 93% (95% CI: 80–98), 88% (71–95) and 79% (62–90) for ovarian, testicular and extragonadal tumours, respectively. The 5-year EFS varied (p =.02) according to the risk groups: 90% (66–97), 64% (30–85), 95% (72–99) and 87% (74–94) for IR1, IR2, HR1 and HR2, respectively. TM decline adjusted to tumour site, and alpha-fetoprotein (AFP) level revealed a prognostic impact of time to normalisation on EFS: HR = 1.03 (1.003–1.007). Conclusion: Risk-adapted and globally decreased chemotherapy burden maintains excellent outcomes, exclusive of the IR2 group, which warrants more intensive chemotherapy

Stratégies préventives et thérapeutiques de la rechute après allogreffe de cellules souches hématopoïétiques : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC)

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Transfert des patients allogreffés de cellules-souches hématopoïétiques en réanimation : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC) [Transfer of allogeneic stem cell transplant recipients to the intensive care unit: Guidelines from the Francophone society of marrow transplantation and cellular therapy (SFGM-TC)]

National audienceTransferring a patient undergoing an allogeneic stem cell transplantation to the intensive care unit (ICU) is always a challenging situation on a medical and psychological point of view for the patient and his relatives as well as for the medical staff. Despite the progress in hematology and intensive care during the last decade, the prognosis of these patients admitted to the ICU remains poor and mortality is around 50 %. The harmonization working party of the SFGM-TC assembled hematologists and intensive care specialist in order to improve conditions and modalities of the transfer of a patient after allogeneic stem cell transplantation to the ICU. We propose a structured medical form comprising all essential information necessary for optimal medical care on ICU

Evaluation of Outcomes and Quality of Care in Children with Sickle Cell Disease Diagnosed by Newborn Screening: A Real-World Nation-Wide Study in France

This study’s objective was to assess, on a national scale, residual risks of death, major disease-related events, and quality of care during the first five years in children diagnosed at birth with sickle cell disease (SCD). Data were retrospectively collected from medical files of all children with SCD born between 2006–2010 in France. Out of 1792 eligible subjects, 1620 patients (71.8% SS or S/beta°-thalassemia -SB°-) had available follow-up data, across 69 centers. Overall probability of survival by five years was 98.9%, with 12/18 deaths related to SCD. Probability of overt stroke by five years in SS/SB° patients was 1.1%, while transcranial Doppler (TCD) was performed in 81% before three years of age. A total of 26 patients had meningitis/septicemia (pneumococcal in eight cases). Prophylactic penicillin was started at a median age of 2.2 months and 87% of children had received appropriate conjugate pneumococcal vaccination at one year. By five years, the probability of survival without SCD-related events was 10.7% for SS/SB° patients. In contrast, hydroxyurea was prescribed in 13.7% and bone marrow transplant performed in nine patients only. In this study, residual risks of severe complications were low, probably resulting from a good national TCD, vaccination, and healthcare system coverage. Nonetheless, burden of disease remained high, stressing the need for disease-modifying or curative therapy

Click-Crosslinked Injectable Gelatin Hydrogels

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Treatment intensity and outcome of nonagenarians selected for admission in ICUs: a multicenter study of the Outcomerea Research Group

International audienceBACKGROUND: Outcome of very elderly patients admitted in intensive care unit (ICU) was most often reported for octogenarians. ICU admission demands for nonagenarians are increasing. The primary objective was to compare outcome and intensity of treatment of octogenarians and nonagenarians. METHODS: We performed an observational study in 12 ICUs of the Outcomerea network which prospectively upload data into the Outcomerea database. Patients \textgreater90 years old (case patients) were matched with patients 80-90 years old (control patients). Matching criteria were severity of illness at admission, center, and year of admission. RESULTS: A total of 2419 patients aged 80 or older and admitted from September 1997 to September 2013 were included. Among them, 179 (7.9 %) were \textgreater90 years old. Matching was performed for 176 nonagenarian patients. Compared with control patients, case patients were more often hospitalized for unscheduled surgery [54 (30.7 %) vs. 42 (23.9 %), p \textless 0.01] and had less often arterial monitoring for blood pressure [37 (21 %) vs. 53 (30.1 %), p = 0.04] and renal replacement therapy [5 (2.8 %) vs. 14 (8 %), p = 0.05] than control patients. ICU [44 (25 %) vs. 36 (20.5 %), p = 0.28] or hospital mortality [70 (39.8 %) vs. 64 (36.4 %), p = 0.46] and limitation of life-sustaining therapies were not significantly different in case versus control patients, respectively. Only 16/176 (14 %) of case patients were transferred to a geriatric unit. CONCLUSION: This multicenter study reported that nonagenarians represented a small fraction of ICU patients. When admitted, these highly selected patients received similar life-sustaining treatments, except RRT, than octogenarians. ICU and hospital mortality were similar between the two groups

Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome

International audiencewas low in thalassemia, normal in SCA, and markedly elevated in MDS (P<0.001). Two mechanisms may explain that iron deposition largely spares the heart in SCA: the high level of erythropoiesis recycles the iron and the chronic inflammation retains iron within the macrophages. Thalassemia, in contrast, is characterized by inefficient erythropoiesis, unable to handle free iron. Iron accumulation varies widely in MDS syndromes due to the competing influences of abnormal erythropoiesis, excess iron supply, and inflammation